DUCHENNE MUSCULAR DYSTROPHY

This study tests a gene therapy called SRP-9001 for Duchenne muscular dystrophy, a severe muscle-weakening disease. About 148 males, both those who can still walk and those who cannot, will receive either the therapy or a placebo. The goal is to see if the treatment improves arm …

This study follows 80 boys with Duchenne muscular dystrophy who have already taken vamorolone in earlier studies. Researchers want to see how safe and effective the drug is over a longer period, especially regarding bone health and fractures. The goal is to better manage the dise…

This study tests an experimental drug called sevasemten in 76 children aged 4-9 with Duchenne muscular dystrophy. The goal is to see if the drug is safe and can help control the disease by measuring side effects, drug levels in the blood, and muscle health markers. Participants a…

This study looks at the long-term safety and effectiveness of a drug called DS-5141b in people with Duchenne muscular dystrophy (DMD). It includes 8 participants who already completed an earlier study of the same drug. Researchers will track side effects and measure changes in mu…

This study tests a gene therapy called PF-06939926 in 114 boys with Duchenne muscular dystrophy (DMD), a severe muscle-weakening disease. Two-thirds of participants receive the gene therapy, while one-third get a placebo (with a chance to switch to the therapy after one year). Th…

This study is for people with Duchenne muscular dystrophy who already took part in the HOPE-2 trial. It tests the long-term safety and effects of a drug called deramiocel, given every three months for about five years. The goal is to see if the drug can help maintain arm and hand…

This study is for people with Duchenne muscular dystrophy who have already taken the experimental drug WVE-N531 in a previous study. The goal is to check the drug's long-term safety and how well it works over time. Participants will receive the drug intravenously and undergo regu…

This early-stage study tests a single intravenous dose of JWK007 in 3 boys aged 5-10 with Duchenne muscular dystrophy (DMD). The main goal is to check safety and side effects, while also looking at whether it helps muscle function. It is a very small, first-in-human trial, so it …

This study tests an experimental drug called EDG-5506 (sevasemten) in 43 children aged 6 to 17 with Duchenne muscular dystrophy who have already received gene therapy. The goal is to see if the drug is safe and how it affects muscle health markers. Participants are randomly assig…

This study follows 66 boys with Duchenne muscular dystrophy who already received RGX-202 gene therapy in an earlier trial. Researchers will monitor safety and track how well the therapy helps with muscle function over time, such as standing, walking, and climbing. The goal is to …

This study tests a drug called TAS-205 in 104 people with Duchenne muscular dystrophy, a muscle-weakening disease. The goal is to see if TAS-205 helps with movement tasks like standing up from the floor and walking, and to check for side effects. Participants are either able to w…

This study tests a single dose of a gene therapy called SGT-001 in 12 boys with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and tolerable. Participants are followed for about 5 years. The therapy is given through a vein and aims to help control the disease…

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to usual steroid treatment. Researchers will track movement, breathing, and safety over time. Participants must already be on or starting steroids, and those ge…

This study tests a gene therapy (delandistrogene moxeparvovec) in children under 4 with Duchenne muscular dystrophy, a severe muscle-weakening disease. The goal is to see if the treatment is safe and helps produce a key muscle protein. About 13 children will take part and be foll…

This study tests an experimental drug called NS-050/NCNP-03 in 20 boys aged 4 to 16 with Duchenne muscular dystrophy (DMD), a genetic condition that causes progressive muscle weakness. The drug aims to help muscles produce a needed protein by skipping a faulty section of the gene…

This study tests a drug called satralizumab in 30 children (ages 8 to 17) with Duchenne muscular dystrophy who are already taking steroids. The goal is to see if the drug can improve bone density and is safe. The study is active but not recruiting new participants.

This study follows 300 people with Duchenne muscular dystrophy who are already taking eteplirsen, golodirsen, or casimersen in regular medical care. Researchers will collect information on movement, breathing, and heart function over time to understand how these treatments work i…

This study looks at how Duchenne muscular dystrophy (DMD) and its treatments affect children's thinking skills (like memory and planning) and their ability to do everyday activities such as self-care, schoolwork, and play. Researchers will compare 38 children with DMD to healthy …

This study follows about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in a previous clinical trial. No new treatment is given. The goal is to monitor long-term safety and how well the therapy continues to work over time. Particip…

This study was designed to track heart muscle changes over time in people with Duchenne muscular dystrophy using cardiac MRI and blood tests. It planned to include children aged 6 and older with a confirmed genetic diagnosis. However, the study was withdrawn before enrolling any …